Pfizer Receives Positive CHMP Opinion for Two Hematology Medicines, MYLOTARG™ and BOSULIF®

MYLOTARG (gemtuzumab ozogamicin) Granted a Positive Opinion for the Treatment of Previously Untreated, De Novo, CD33-positive Acute Myeloid Leukemia in Combination with Chemotherapy

BOSULIF (bosutinib) Granted a Positive Opinion for the Treatment of Newly Diagnosed Ph+ Chronic Myelogenous Leukemia

NEW YORK, NY, USA I February 23, 2018 I Pfizer Inc. (NYSE:PFE) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted positive opinions recommending that two Pfizer hematology medicines be granted marketing authorizations in the European Union (EU). MYLOTARG™ (gemtuzumab ozogamicin) in combination with daunorubicin and cytarabine has been granted a positive opinion for the treatment of patients age 15 years and above with previously untreated, de novo, CD33-positive acute myeloid leukemia (AML), except acute promyelocytic leukemia (APL). BOSULIF® (bosutinib) has been granted a positive opinion for the treatment of adults with newly diagnosed chronic phase Philadelphia chromosome-positive chronic myelogenous leukemia (Ph+ CML). The CHMP’s opinions for both medicines will now be reviewed separately by the European Commission (EC).

“There is an urgent need to improve outcomes for leukemia patients in Europe,” said Mace Rothenberg, M.D., chief development officer, Oncology, Pfizer Global Product Development. “If approved, the addition of MYLOTARG to standard chemotherapy will provide an important new treatment option for patients with acute myeloid leukemia who would typically be treated with chemotherapy alone. Additionally, the potential expansion of the approved use of BOSULIF to include first-line therapy expands the treatment options for adult patients with newly diagnosed chronic myelogenous leukemia.”

The Marketing Authorization Application (MAA) for MYLOTARG was based on data from an investigator-led, Phase 3, randomized, open-label study (ALFA-0701) in previously untreated, de novo patients.

BOSULIF currently has conditional marketing authorization in Europe related to the initial marketing authorization. The Type II Variation application for BOSULIF for adults with newly diagnosed chronic phase Ph+ CML was based on results from BFORE (Bosutinib trial in First line chrOnic myelogenous leukemia tREatment), a randomized multicenter, multinational, open-label, Phase 3, head-to-head study of BOSULIF 400 mg versus imatinib 400 mg, a current standard of care.

Pfizer and Avillion entered into an exclusive collaborative development agreement in 2014 to conduct the BFORE trial. Under the terms of the agreement, Avillion provided funding for the trial to generate the clinical data used to support this application and other potential regulatory filings for marketing authorization for BOSULIF as first-line treatment for patients with chronic phase Ph+ CML. Pfizer retains all rights to commercialize BOSULIF globally.


Acute myeloid leukemia is a rapidly progressing, life-threatening blood and bone marrow cancer. 1 If left untreated, patients with AML will die within months, if not weeks, of their disease. AML is the most common type of acute leukemia in adults and accounts for approximately 80% of all cases of acute leukemia.2 About 1/33,000-1/25,000 people are expected to be newly diagnosed with AML in Europe annually.2


Chronic myelogenous leukemia (CML) is a rare blood cancer, which begins in the bone marrow, but often moves into the blood.3 Researchers estimate that by 2020, more than 412,000 people worldwide will be diagnosed with leukemia (all types).4 Across Europe, CML constitutes about 15% of all leukemia and occurs with an incidence of about 1-1.5/100,000.5

About MYLOTARG™ (gemtuzumab ozogamicin)

MYLOTARG is an antibody-drug conjugate (ADC) composed of the cytotoxic agent calicheamicin, attached to a monoclonal antibody (mAB) targeting CD33, an antigen expressed on the surface of myeloblasts in up to 90 percent of AML patients.6,7,8 When MYLOTARG binds to the CD33 antigen on the cell surface it is absorbed into the cell and calicheamicin is released causing cell death.7,8

MYLOTARG was approved by the U.S. Food and Drug Administration in September 2017 for adults with newly diagnosed CD33-positive AML, and adults and children 2 years and older with relapsed or refractory CD33-positive AML. MYLOTARG was originally approved in 2000 at a higher dose under the FDA’s accelerated approval program for use as a single agent in patients with CD33-positive AML who had experienced their first relapse and were 60 years or older and who were not considered candidates for other cytotoxic chemotherapy. In 2010, Pfizer voluntarily withdrew MYLOTARG in the U.S. after a confirmatory trial failed to show clinical benefit and there was a higher rate of fatal toxicity compared to chemotherapy. MYLOTARG has been available to individual patients through Pfizer’s compassionate use programs.

In addition, MYLOTARG is commercially available in Japan where it has been approved since 2005 for the treatment of patients with relapsed or refractory CD33-positive AML who are not considered candidates for other cytotoxic chemotherapy.

MYLOTARG originates from a collaboration between Pfizer and Celltech, now UCB. Pfizer has sole responsibility for all manufacturing, clinical development and commercialization activities for this molecule.

Pfizer also collaborated with SFJ Pharmaceuticals Group on the registrational program for MYLOTARG.

ABOUT BOSULIF® (bosutinib)

BOSULIF® (bosutinib) is an oral, once-daily, tyrosine kinase inhibitor (TKI), which inhibits the Bcr-Abl kinase that promotes CML; it is also an inhibitor of Src-family kinases. In the U.S., BOSULIF (bosutinib) is indicated for the treatment of adult patients with newly-diagnosed chronic phase Philadelphia chromosome-positive chronic myelogenous leukemia (Ph+ CML). Continued approval for this indication may be contingent upon verification and confirmation of clinical benefit in an ongoing long-term follow up trial. BOSULIF is also indicated in the U.S for the treatment of adult patients with chronic, accelerated or blast phase Ph+ CML with resistance or intolerance to prior therapy (first approved in September 2012).

In Europe, BOSULIF was granted conditional marketing authorization in March 2013 for the treatment of adult patients with Ph+ CML previously treated with one or more TKIs and for whom imatinib, nilotinib and dasatinib are not considered appropriate treatment options.

About Pfizer Oncology

Pfizer Oncology is committed to pursuing innovative treatments that have a meaningful impact on people living with cancer. Our growing pipeline of biologics, small molecules, and immunotherapies is focused on identifying and translating the best scientific breakthroughs into clinical application for patients across a diverse array of solid tumors and hematologic cancers. Today, we have 10 approved oncology medicines and 17 assets currently in clinical development. By maximizing our internal scientific resources and collaborating with other companies, government and academic institutions, as well as non-profit and professional organizations, we are bringing together the brightest and most enterprising minds to take on the toughest cancers. Together we can accelerate breakthrough treatments to patients around the world and work to redefine life with cancer.

Pfizer Inc.: Working together for a healthier worldTM

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world's best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at In addition, to learn more, please visit us on and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at

1 The portal for rare diseases and orphan drugs. Accessed February 2018.

2 Leukemia & Lymphoma Society, Acute Myeloid Leukemia Booklet. Developed 2011. Accessed February 2018.

3 American Cancer Society. What is Chronic Myeloid Leukemia? Accessed February 2018.

4 GLOBOCAN Online Analysis/Prediction. Accessed February 2018.

5 European Treatment and Outcome Study. Accessed February 2018.

6 Griffin JD, Linch D, Sabbath K, et al: A monoclonal antibody reactive with normal and leukemic human myeloid progenitor cells. Leuk Res. 8: 521-534, 1984 CrossRefMedline.

7 Tanaka M, Kano Y, et al. The cytotoxic effects of gemtuzumab ozogamicin (Mylotarg) in combination with conventional antileukemic agents by isobologram Analysis In Vitro. Anticancer Research. 2009; 29: 4589-4596.

8 O’Hear C, Heiber JF, Schubert I, Fey G, Geiger TL. Anti-CD33 chimeric antigen receptor targeting of acute myeloid leukemia. Haematologica. 2015;100(3):336-344.

SOURCE: Pfizer

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